Abstract: Transient transfection-based AAV manufacturing continues to face an industry-wide yield ceiling, limiting scalability, increasing costs, and challenging the commercial viability of gene therapy programs.
AAV vectors offer clear benefits for gene therapy, and continued advances in technology have increased target specificity, improved potency, and intensified production efficiency.
AAV gene therapy programs face long development cycles, raw material variability, and complex scale-up demands.
Summary: Cell and gene therapies continue to expand across oncology, cardiovascular, and CNS diseases, yet production remains limited by talent shortages, complex supply chains, and slow development cycles.
AAV genomic integrity is based on defining the molecular DNA composition of recombinant AAV vector preparations, specifically, the presence of full-length, ITR-to-ITR genomes, and the relative amount of undesirable molecular sub-species encapsidated within AAV particles.
Plasmid DNA is a vital component in the production of viral and nonviral cell and gene therapies (CGT). Catalent’s innovative UpTempo℠ Plasmid Platform Process has revolutionized plasmid DNA manufacturing by addressing key challenges and significantly reducing development time.
